Bladder Cancer Patient with Rare mTOR Mutations Shows Exceptional Drug Response

A patient with advanced bladder cancer experienced a complete response for 14 months to the drug combination everolimus and pazopanib in a phase I trial, and genomic profiling of his tumor revealed two alterations that may have caused this exceptional response, according to a study published onine on March 13, 2014 in Cancer Discovery, a journal of the American Association for Cancer Research (AACR). This information can help identify cancer patients who may respond to everolimus. Exceptional responders are cancer patients who had a complete response or partial response for at least six months to treatment in a clinical trial in which less than 10 percent of patients responded, according to the National Cancer Institute. "Studying exceptional responses can help us understand the specific reasons why some tumors are highly sensitive to certain anticancer agents," said Nikhil Wagle, M.D., an instructor in medicine at Dana-Farber Cancer Institute, and an associate member at the Broad Institute in Cambridge, Massachusetts. "We can use that information to identify patients whose tumors have genetic alterations similar to those found in exceptional responders, and treat them with those same agents. "We conducted a phase I clinical trial of two anticancer agents—the mTOR inhibitor everolimus, and pazopanib, another drug used to treat kidney cancer—and one of our patients developed near complete remission of his bladder cancer which lasted for 14 months," said Dr. Wagle.
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